Revision of the EU general pharmaceuticals legislation
Short Statement from the European Infrastructure for Translational Medicine (EATRIS)
Drug repurposing holds great promise to meet many of the unmet needs that still afflict patients across Europe. Repurposing, the process whereby existing licensed medicines are developed as treatments for diseases for which they are not currently approved, applies across all disease areas, from rare diseases to cancer to the COVID-19 pandemic. However, the full potential of repurposing may stay unrealised because there is often a tension between the needs of patients and the commercial drives of the pharmaceutical industry. Proving the efficacy of a drug in a new disease is a costly endeavour that requires substantial investment and consequently stakeholders expect a return on that investment. This system disadvantages repurposing opportunities for rare and ultra-rare disease populations and for diseases requiring shorter treatment periods compared to longer, for example neo-adjuvant versus adjuvant treatment in oncology.
When a repurposed drug is a widely available generic, the commercial producers often lack incentives to fund that clinical development since their profit margins are more limited (for example, in the Netherlands a 2021 report stated that 50% of generics had a turnover of only 50.000 Euro per year, leaving no margin to reinvest in R&D) and furthermore other companies could free-ride on their investment.
Thus, even when supportive data are produced via public or philanthropic funding there is often no commercial interest in seeking regulatory approval for the drug to treat the new disease. Without official approval, patients are often not able to access the repurposed drug because prescribers and health systems tend to be resistant towards the use of the drug off-label; i.e., the use of a medicine for an unapproved indication or in an unapproved age group, dosage, or route of administration. This situation is a key barrier to successfully making new treatments available to patients across Europe.
Currently, only a marketing authorisation holder (company with a license to market the drug) can apply for approval of a drug to treat a new disease, a process known as a label extension. This becomes an obstacle in those repurposing cases where no commercial rationale exists for a company to make the investment required, even if there are already new data supporting the repurposing for the new indication. The advantages of on-label repurposing are manifold: scientific evidence is officially evaluated, patients’ and physicians’ concerns are addressed, inequities are reduced and reimbursement across all EU members states is promoted. Furthermore, the risks that lead to post marketing authorisation issues in relation to liabilities, drug supplies and data collection are reduced.
We welcome that this issue is being addressed in the Commission proposal for the Pharmaceutical Regulation (dated 26 April 2023). Finding a mechanism that can support a pathway to label extension not solely dependent on commercial considerations, and respecting the needs of all stakeholders, is a vital step forward. In this way patients can gain access to repurposed drugs on approval, public and philanthropic funders are assured that the successful trials they fund can lead to clinical adoption and companies will be able to market their products for the new therapeutic uses.
Scientific and medical innovation increasingly needs policy innovation before it can make a positive difference to health and well-being. As a European research infrastructure, working with clinicians, researchers, philanthropic funders and industry, we strongly support any initiatives that will accelerate the translation of research for the benefit of patients.
Disclaimer
This statement exclusively reflects the views and opinions of the scientific leadership of the EATRIS Coordination and Support Office, headquartered in Amsterdam, The Netherlands, and do not necessarily reflect the official position of EATRIS governmental and institutional members or partners in EATRIS-led initiatives.
Who is EATRIS?
EATRIS is a not-for-profit organisation currently supported by 14 European member states, with headquarters based in Amsterdam (The Netherlands). We regroup 150 academic research institutes and university medical centres, collectively driven to accelerate the translation of scientific discoveries into patient benefit. We focus our activities on early stages of research (pre-clinical, early clinical) where the risk of failure is particularly high and the need for multi-stakeholder engagement crucial. www.eatris.eu
Our main objectives include:
- Foster public-private partnerships: we initiate and facilitate academia-industry collaborations between SME, large pharma and EATRIS member institutes.
- Overcome barriers to health innovation: we offer access to regulatory, intellectual property, product development and health technology assessment expertise. We also support funders of translational research through quality assessment and mentoring programmes, ensuring their investment leads to limited funding waste and optimised patient impact.
- Train the next generation of translational scientists and innovation leaders: we develop training programmes for early-career researchers in translational research with specific attention paid to exposure to non-academic sector, transversal skills and translational gaps.
EATRIS currently leads a 25 Mi Euros Horizon Europe-funded initiative, REMEDi4ALL, to build a sustainable European innovation platform for medicines repurposing. Find out more: https://remedi4all.org/
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Contact
- Donald Lo, EATRIS Director of Medicines Development | donaldlo@eatris.eu
